List of diseases with a known mechanism but no cure/treatment

[Utsikt]

I don't quite understand. If you have an intractable disease, you can either wait for the science to find the solution or do something on your own. Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

There is if you’re asking someone to help you get the medications.

It’s a bit like the assisted suicide debate in that specific regard.

 

[Trish]

I don't quite understand. If you have an intractable disease, you can either wait for the science to find the solution or do something on your own. Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

This disregards the prescribing doctor's professional ethics and insurance. A responsible doctor is surely unlikely to risk their career and reputation by prescribing unevidenced drugs for a patient to try without being provided with some evidence to support that experiment.

 

[Kitty]

Why shouldn't your belief effect your choice? It's just a matter of evaluating the odds according to your belief; there is no social/medical ethics there as far as I can see.

Because it's obviously not about me. It's a much bigger ethical issue, and I'm not a special case who can be exempted from it.

As soon as doctors start experimenting on patients—and they would be doing that, prescribing drugs makes them active participants—the whole ethical paradigm begins to break down. Thin end of a wedge and all that.

 

[Tree]

But what about the ethical issue of abandoning, and refusal of funding biomedical research into the disease of very ill people? Even worse forcing people to undergo treatments that make them worse?

Isn’t very cruel to expect the very ill patients to wait for biomarker research, trials when the funding that is needed has not come in the last 30 years and will not likely come in the next few years?

To expect people not to try anything, is almost just as unethical.

The issue is not as black and white, in regard to medications being unsafe
- if there are medications that are safe for human use
- if these medications are of low toxicity and few adverse effects
- if these medications have been tested as safe for similar complaints

It would not be like shooting in the dark, because testing has been there.

Than these medications should be available for the individual patient who wants to, to carefully try.

 

[Kitty]

But what about the ethical issue of abandoning, and refusal of funding biomedical research into the disease of very ill people? Even worse forcing people to undergo treatments that make them worse?

Yes, I do think it's unethical. But two wrongs don't make a right.

Isn’t very cruel to expect the very ill patients to wait for biomarker research, trials when the funding that is needed has not come in the last 30 years and will not likely come in the next few years?

Of course, but the same applies to many causes of disease and disability. In some cases it probably won't be a wait of a few years to get solutions, it could be a few lifetimes.

I've lived with ME/CFS for 49 years without any help, and a relative lived with it for 73 years. The fact we were unlucky enough live in a time before there was a treatment—just as her brother was unlucky enough to live in a time when there was no treatment for Type 1 diabetes, so he died in pain at the age of four—feels cruel, but it's how it goes.

I struggle to think about it without considering the wider context, and that is that we won't improve science by allowing doctors to experiment on people outside of clinical trials. We'll just erode the ethical framework further by allowing the existing wild west situation to gain momentum, and reward them for ever-diminishing standards. Our children and grandchildren won't thank us for that legacy, and they certainly won't benefit from it.

I understand your perspective and I know how much people need progress to be made on treating ME/CFS, but at the same time I can't un-understand that it's not only about us. I hope that makes some kind of sense, anyway.

 

[poetinsf]

It’s a bit like the assisted suicide debate in that specific regard.

Is the off-level use of safe drugs really comparable to assisted suicide? Or is it just your belief? If it is your belief, is it fair to impose that on others? The ethics here is no different than any other off-level use of safe drugs. The only difference is the possibility of making your ME/CFS permanently worse. That, as I said before, is as much a belief as improving permanently.

 

[Utsikt]

Is the off-level use of safe drugs really comparable to assisted suicide? Or is it just your belief? If it is your belief, is it fair to impose that on others? The ethics here is no different than any other off-level use of safe drugs. The only difference is the possibility of making your ME/CFS permanently worse. That, as I said before, is as much a belief as improving permanently.

I did not say it was comparable to assisted suicide in general.

I said it was comparable to assisted suicide in that regard, in context of the previous sentence that referenced the ethical considerations in play when another person has to act in order for you to be able to test a treatment.

A person with a medical, legal and ethical responsibility to protect your wellbeing has to agree do let you try something that has the potential to cause harm. They will bear the personal responsibility if something goes wrong.

Therefore, the choice to try the restricted treatments can’t be entirely yours. You can’t force them to write a prescription if they don’t want to.

And by extension of that, the choice falls to the society through their governing mechanisms, through which they decide the legal, medical and ethical framework in which the practitioner is allowed to make a judgement about what they will and won’t allow you to try.

 

[Covidivici]

"You're all wrong. Welcome to Long COVID"
Has been my catch phrase for the past few years.
Or in this case, I think you're all right. I find myself agreeing with every opposing argument. Because of the big black void.
The way I break it down is thus:

• Until we can measure this condition, beyond patient self-reporting, we won't be able to evaluate treatment efficacy, beyond patient self-reporting. That's entirely too subjective to my liking. Too many moving parts.
• Until we know what's behind the dysfunction, any potential treatment will remain a stab in the dark. The odds of hitting the target are not just slim — they are practically null.

However:

• Serendipity hasn't only led to some medical breakthroughs, but a plurality of civilization-altering findings. And you aren't going to hit a damn thing unless you keep stabbing.

So I say we keep stabbing. While also searching for the underlying mechanisms. It isn't a zero-sum game, as most of the blind stabbing is being done by patient-led initiatives supported by trusted doctors who are often (as in my case) friends. Predatory quacks are another matter altogether. They deserve censure, prison time and bankruptcy. But I digress.

If a patient (me) asks for a trial of some molecule that makes at least a minimum of physiological sense, has no contraindications, and appears to have benefited some people (which means less and less to me as time goes on — those same people tend to reappear on the Long Hauler forums months later, conveniently forgetting that their recovery story should probably undergo some editing/updating), why the hell not try?

Because it could make me worse? That due diligence is on me and my attending to suss out beforehand and for the duration of treatment. Is it a waste of time and money? Probably. Any more than just rotting away with this disability? Not if it yields some positive results. I used to get a lot done.

Which leads me back to the reason I agree with both sides in this debate: we don't know. Probabilities are one thing, but sheer dumb luck has cured more than a few intractable diseases. Place your bets. May the best option win.

You’re essentially asking a mechanic to try and fix a car by going to a general hardware store and picking an item randomly.

I love this analogy. Stealing it.

I disagree. The difference is not in our perception of how likely ‘basic science’ is to produce a result. It’s in how likely it is that ‘trying promising drugs’ will produce a result.

IMO that chance is essentially zero.

You're probably right, but time will tell — cause you ain't stopping me!

Thinking through this again, I believe me and @poetinsf disagree on both how likely it is basic science will find a cure and for random experiments to find a cure.

Hard disagree. Just look at how successful alchemists were at turning stuff into gold! Wait... Oh.

I believe random experiments have a near zero chance of finding a cure. And that basic science has a substantially higher chance.

NEAR zero. So there is a chance! (But yes, basic science is the light switch. By all means, turn on the lights).

As far as I can tell, nobody except for maybe the person that wrote the compiler, knows how computer programs really work. They frequently crash, require constant maintenance and often just do the bare minimum they have to do in ways that really were not intended. When you fix a bug, you often have no idea why it occurred in the first place, and even less of an idea of why the new thing worked.

The human body is so much more complex that it would be exceedingly unlikely to stumble upon any useful information or insigth by trying things randomly or even semi-informed.

We can’t even figure out how AI models work, and we created them. They are far less complex than the human body.

THANK YOU! This AI bubble isn't just a bubble because of speculative investing, but because so few people understand the technology's fundamental limitations.

"OpenAI admits AI hallucinations are mathematically inevitable, not just engineering flaws." —ComputerWorld Sept 18th, 2025

You could have 5 people that say that they feel good after doing CBT for a week or you could have 5 people that report feeling horrible, we know there are plenty reasons for either and we know why neither matter. How would you select what constitutes a success when you're purely relying on subjective reports were some people might be more prone to believe in something rather than something else and you'd likely end up picking those that are more likely to believe in something?

Exactly. As I said: too fuzzy. Too unreliable.

It is of course plausible that one can stumble upon a treatment via luck, similar to how things happened with Rituximab, even though it didn't work in the end, but I don't see this approach being a very fruitful one at getting there but there are probably meaningful approaches how one could increase ones luck (for example by setting up long-term patient registers).

Eureka Health seemed promising as a repository of compiled accounts as to the effectiveness (or lack thereof) of treatments, but the platform pivoted to AI slop. There are such things as https://longhaulwiki.com/treatment-outcomes/ (which I'm not familiar with, was looking for Eureka just now when I saw they had flushed all their data and saw Long Haul Wiki). I'm thinking something a little more formalized would be to everyone's benefit. Instead of everyone not only stabbing in the dark, but without the benefit of everyone else's experience in stabbing here vs there.

Anyway. Onward. We'll get there.

 

[Utsikt]

Until we can measure this condition, beyond patient self-reporting, we won't be able to evaluate treatment efficacy, beyond patient self-reporting. That's entirely too subjective to my liking. Too many moving parts.

It done properly and for long enough, there are some objective outcomes that could be used like ability to work/go to school, time spent upright (if OI is an issue) and step count. But that would require a really good sense of baseline measurements to ensure that the patients don’t just replace activity X with activity Y (without any net benefit) and that the effect is sustained.

So I say we keep stabbing. While also searching for the underlying mechanisms. It isn't a zero-sum game

If you account for the negative outcomes it’s probably a net negative game to stab.

If a patient (me) asks for a trial of some molecule that makes at least a minimum of physiological sense

How would you determine what makes sense when we don’t know much about what’s going on?

Which leads me back to the reason I agree with both sides in this debate: we don't know. Probabilities are one thing, but sheer dumb luck has cured more than a few intractable diseases.

Those attempts have also killed very many people. And wasted a lot of resources which has prolonged the suffering of everyone because the actual solution took longer.

 

[Utsikt]

There are such things as https://longhaulwiki.com/treatment-outcomes/ (which I'm not familiar with, was looking for Eureka just now when I saw they had flushed all their data and saw Long Haul Wiki)

That list was interesting. Brain retraining as #3 and prayer as #4. Which might tell you something about all the treatments below those!

 

[Covidivici]

That list was interesting. Brain retraining as #3 and prayer as #4. Which might tell you something about all the treatments below those!

Full disclosure, I hadn't even peaked at the list before posting the link (wife woke up and came down to discuss the draconian law being passed by our provincial gvt, so I just posted reply). But what you report definitely checks out with what I've been reading on Reddit (let alone Facebook, which I left years ago in protest).

I guess I'm also suffering from a form of recency bias: I know SARS CoV-2 is what triggered this in me. So it's easy (if erroneous) to believe that we might be able to pinpoint the "how" relatively quickly (by looking into the mechanisms the virus upended/corrupted.

As for the fatalities in previous failed ME treatment attempts, I have to plead ignorance as it wasn't on my radar prior to the pandemic (other than the disastrous push for GET I've read about). But I'm guessing they were fewer than those still being killed, maimed and brain damaged by COVID. That's the clue I'm hoping will elucidate the mechanism behind the pathology (and why I still think the Putrinos of the world aren't just wasting time).

But I do see your point. And you're right that stabbing blindly and without some organized strategy can lead to more than just frustration.

 

[Utsikt]

As for the fatalities in previous failed ME treatment attempts, I have to plead ignorance as it wasn't on my radar prior to the pandemic (other than the disastrous push for GET I've read about).

I don’t know about direct fatalities from experimental treatments in ME (I have not looked for them), but experimenting with drugs in general has lead to many fatalities.

Fatalities and negative effects in general are also under-reported. Jonathan Edwards almost had to sue someone to get to know about a death in his own trial.

Post in thread 'Teclistamab for ME/CFS'

Aug 4, 2025

Could you elaborate? As far as I know, I don't know what data they have nor what diagnostics, inclusion criteria etc they use. If done properly, why would it not generate useful data? Genuinely curious.

The first thing you want to know is improvement in the condition but for ME/CFS the measures are so subjective and placebo and relate issues so big that you are not going to get any direct data. There are not going to be any indirect or surrogate data because there are no abnormalities like raised CRP that we know to be surrogates. There are no known relevant autoantibodies so they...

• Jonathan Edwards

• 

The page you linked also mentions a fatality in LC from apheresis.

 

[Creekside]

I love this analogy. Stealing it.

I expect there are plenty of stories of at least temporary vehicle repairs made with random items. If your fuel pump fails, you could drive home by rigging up a container to gravity-feed fuel into a carburetor. A lot of those examples are pre-vehicle-computer days, and pre-cellphone-to-call-for-tow days.

I don’t know about direct fatalities from experimental treatments in ME (I have not looked for them), but experimenting with drugs in general has lead to many fatalities.

For this debate, we are ignoring the fatalities due to experimenting with risky drugs. Covidivici said "no contraindications". My doctor was willing to write a prescription for LDN, because he was convinced that it was low-risk ... and it ended up working really well for me! I even managed to get some riskier drugs (cyclosporin) because I was desperate. I wonder how many PWME have committed suicide because they lost hope after trying
even a safe drug was refused.

It's not trying anything vs banning all experiments; it's a request for the option of trying something relatively safe compared to the risk of trying nothing. How many severe cases of ME might have been prevented if they'd been allowed to experiment a bit more? No easy answers.

My perspective is that I have experimented, and had several valuable successes and no long-term failures. Lotteries offer only tiny chances of success, but plenty of people buy tickets. The same for other forms of gambling, with significant chances of long-term harm (divorce, bankruptcy, deep debt to criminals).

 

[Utsikt]

For this debate, we are ignoring the fatalities due to experimenting with risky drugs. Covidivici said "no contraindications".

I think you should go back and read JE’s comments. Most drugs are not safe.

I wonder how many PWME have committed suicide because they lost hope after trying
even a safe drug was refused.

That’s a very distasteful speculation in my opinion. If you want to talk about sources of despair, why not mention the negative consequences of repeated failures of treatments that were tried on false premisses due to irresponsible clinicians willing to prescribe them? Severely ill patients being forced to experiment by those around them because they think it can never cause harm. By delayed adaptation to their illness because this is just temporary and surely I’ll find the solution soon enough. Or the added burden from financial strain. The constant barrage of unhelpful suggestions.

All because experimenting has become normalised.

It's not trying anything vs banning all experiments; it's a request for the option of trying something relatively safe compared to the risk of trying nothing. How many severe cases of ME might have been prevented if they'd been allowed to experiment a bit more? No easy answers.

Again, you’re only looking at one side of the equation.

 

[Mij]

My best friend died from ME complications, he tried almost every drug available that made so called claims from high profile ME/CFS doctors who had their own theories. People are very ill and desperate and will take chances on unproven treatments.

 

[Creekside]

Most drugs are not safe.

Yes, but I'm certainly not saying that we should be trying "most" drugs. Nothing is 100 safe, including water or sucrose. Some drugs are highly likely to cause harm, especially when taken long-term. Other drugs are relatively safe (safer than some common foods), especially if taken for only a few doses. The problem is defining what is too risky to be worth trying.

A few PWME have regretted trying LDN. I get the impression that more PWME who have tried it had gotten worthwhile benefits. So, should trying LDN be outright banned, or allowed? I think that's "looking at both sides of the equation".

 

[Utsikt]

A few PWME have regretted trying LDN. I get the impression that more PWME who have tried it had gotten worthwhile benefits. So, should trying LDN be outright banned, or allowed? I think that's "looking at both sides of the equation".

You will always have more positive than negative anecdotes for a treatment if the treatment doesn’t do much, due to loads of mechanisms and biases that controlled trials try to get around.

We have no reliable evidence for using LDN as a treatment for ME/CFS. There are multiple ongoing trials for ME/CFS and/or LC. LDN has failed when trialled for FM.

In sum, LDN should not be prescribed for ME/CFS, LC or FM outside the clinical trials, and the rationale for doing the trials is shaky at best.

 

[Sean]

One of the reasons for Helsinki protocols, et al, is because desperate patients are easy targets for exploitation.

They exist for a very good reason. Safety rules are written in blood.

 

[shak8]

You will always have more positive than negative anecdotes for a treatment if the treatment doesn’t do much, due to loads of mechanisms and biases that controlled trials try to get around.

We have no reliable evidence for using LDN as a treatment for ME/CFS. There are multiple ongoing trials for ME/CFS and/or LC. LDN has failed when trialled for FM.
(
In sum, LDN should not be prescribed for ME/CFS, LC or FM outside the clinical trials, and the rationale for doing the trials is shaky at best.

What made me throw away the LDN capsules I ordered (not ingesting even one) is that the compounding pharmacy's record of inspection (where my capsules were made) showed unsanitary practices and they found capsules not filled correctly (yielding low dose).

My concern with compounding pharmacies is that they are not fully inspected by government agencies.

This is my same concern with supplements. You aren't assured or reassured of the manufacturing process, the ingredients, and the dosage.

It's a risk/benefit intuition and I'm risk averse. I prefer waiting until a drug is well past the post-marketing (phase 4) state to elicit more adverse reactions. And then there are more studies confirming or not, the benefit of the drug. All this takes too much time, alas.

I try to remember the underlying profit motive in heatlhcare that seems an equal player with altruism.

 

[Creekside]

In sum, LDN should not be prescribed for ME/CFS,

I for one am very grateful that my GP was willing to write a prescription for it. It made a huge improvement in my quality of life. I don't regret any of the prescriptions I tried. I haven't tried an new ones in a long time, but that's because I haven't found any I've considered any more likely to work than plant parts selected at random.